South Korean biotech ENCell Co., Ltd. has reported promising results from its first-in-human Phase 1 clinical trial of EN001, a novel mesenchymal stem cell therapy for Charcot-Marie-Tooth disease type 1A (CMT1A). The results were presented at the Peripheral Nerve Society annual meeting in Montreal on June 23, 2024, marking a significant milestone for patients with this rare neurological condition who currently have no approved treatment options.
Trial Design and Safety Profile
The Phase 1 trial enrolled nine CMT1A patients at Samsung Medical Center, with Professor Byung-Ok Choi from the Department of Neurology serving as Principal Investigator. The study utilized a dose-escalation design to evaluate the safety and exploratory efficacy of EN001 over a 16-week period, completing in June 2023.
The trial demonstrated a favorable safety profile, with no dose-limiting toxicity, serious adverse events, or infusion-related responses observed among participants. This safety confirmation represents a crucial first step for EN001, which is developed using ENCell's proprietary technology and designed as a next-generation allogeneic mesenchymal stem cell therapy.
Efficacy Outcomes Show Significant Improvements
The exploratory efficacy evaluations revealed statistically significant improvements in disease severity measures. The Charcot-Marie-Tooth Neuropathy Score version 2 decreased by an average of 2.89 points (p=0.0039) after 16 weeks across all participants. Notably, the high-dose group demonstrated a more pronounced reduction of 3.50 points (p=0.0313).
Further analysis indicated that patients receiving the high dose experienced meaningful clinical improvements in disease severity, with some patients transitioning from severe to moderate categories or from moderate to mild categories. The high-dose group also showed marked improvements in specific sensory and motor function assessments.
Functional Improvements Across Multiple Measures
The trial documented enhancements across several functional assessment tools, including the 10 Meter Walking Test, Functional Disability Scale, and Overall Neuropathy Limitation Score leg scale. Nerve conduction studies also showed improvements, particularly in the high-dose group, suggesting potential neurological benefits from the stem cell therapy.
According to ENCell's non-clinical studies, EN001 can migrate to and regenerate damaged nerves and muscles, which may explain the observed functional improvements in trial participants.
Clinical Significance and Future Plans
Professor Byung-Ok Choi expressed optimism about the results, stating, "These results provide hope to CMT1A patients, a group affected by a rare disease without existing treatments." The significance of these findings is amplified by the current lack of approved therapies for CMT1A, a hereditary peripheral neuropathy that affects nerve function and muscle strength.
An ENCell spokesperson emphasized the company's dedication to developing new treatment options for CMT patients, highlighting the importance of presenting these clinical results at the prestigious Peripheral Nerve Society conference, a key gathering for neurologists, neurosurgeons, neuroscientists, and other healthcare professionals focused on peripheral nerve disorders.
ENCell is planning to conduct a repeat-administration clinical trial at Samsung Medical Center, building on these encouraging Phase 1 results to further evaluate the therapeutic potential of EN001 in CMT1A patients.
